FDA Approves First Treatment for Patients with Plasminogen Deficiency, a Rare Genetic Disorder
"Today's approval helps address an unmet medical need for individuals affected by this rare genetic disease."
- "Today's approval helps address an unmet medical need for individuals affected by this rare genetic disease."
- Individuals with this disease lack a protein called plasminogen, which is responsible for the ability of the body to break down fibrin clots.
- Treatment with Ryplazim helps to increase the plasma level of plasminogen - enabling a temporary correction of the plasminogen deficiency and reduction or resolution of the lesions.
- The FDA granted Ryplazim Orphan Drug designation, which provides incentives to assist and encourage drug development for rare diseases.